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Somapacitan in children born small for gestational age: a multi-centre, open-label, controlled phase 2 study

Anders Juul1,2, Philippe Backeljauw3, Michael Højby4

  • 1Department of Growth and Reproduction, Copenhagen University Hospital - Rigshospitalet, Copenhagen 2100, Denmark.

European Journal of Endocrinology
|January 18, 2023
PubMed

Insights

Once-weekly somapacitan demonstrated comparable efficacy, safety, and tolerability to daily growth hormone (GH) in children born small for gestational age (SGA). The 0.24 mg/kg/week dose showed the most promise for treating short stature in these children.

Area of Science:

  • Pediatric endocrinology
  • Growth disorders
  • Pharmacology

Background:

  • Short stature in children born small for gestational age (SGA) is a significant clinical concern.
  • Current treatment involves daily growth hormone (GH) injections, which can impact adherence and quality of life.
  • Novel therapeutic strategies, such as once-weekly somapacitan, aim to improve treatment outcomes and patient experience.

Purpose of the Study:

  • To evaluate the efficacy, safety, and tolerability of three different once-weekly doses of somapacitan.
  • To compare these somapacitan doses against standard daily GH administration in prepubertal children born SGA.
  • To identify the optimal somapacitan dosage for this patient population.

Main Methods:

  • A randomized, multi-center, open-label, controlled phase 2 study was conducted.
  • Sixty-two treatment-naïve, prepubertal children born SGA were randomized to receive either once-weekly somapacitan (0.16, 0.20, or 0.24 mg/kg/week) or daily GH (0.035 or 0.067 mg/kg/day) for 26 weeks.
  • Height velocity (HV), height standard deviation score (SDS), and insulin-like growth factor I (IGF-I) SDS were assessed.

Main Results:

  • All somapacitan doses and daily GH doses demonstrated dose-dependent increases in HV, height SDS, and IGF-I SDS.
  • The highest somapacitan dose (0.24 mg/kg/week) achieved an annualised HV of 11.3 cm/year, comparable to daily GH (0.067 mg/kg/day) at 11.9 cm/year.
  • Exposure-response modeling indicated that higher somapacitan exposure correlated with greater efficacy.
  • Safety and tolerability profiles were similar across all treatment groups.

Conclusions:

  • Once-weekly somapacitan, particularly at 0.24 mg/kg/week, shows comparable efficacy, safety, and tolerability to daily GH 0.067 mg/kg/day in short children born SGA.
  • Somapacitan offers a potential alternative to daily GH injections, possibly improving treatment adherence.
  • Further long-term studies are warranted to confirm these findings.
Abstract

Keywords:
growth hormonelong-acting growth hormonesmall for gestational agesomapacitan

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